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Freeline presents preclinical liver-directed AAV gene therapy data at the European Working Group on Gaucher Disease (EWGGD)

London, 5 July, 2019 – Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, will present preclinical data on its gene therapy development candidate for Gaucher Disease at the European Working group on Gaucher Disease (EWGGD) in Auvergne, France, July 4-6 2019. The Freeline program FLT200  is a liver-directed Adeno-associated virus (AAV)  gene therapy  for the treatment of  type 1 Gaucher Disease and  is designed to increase levels of beta-glucocerebrosidase (GCase) in macrophages of Gaucher’s Disease patients.  

Gaucher Disease is a genetic disorder in which a fatty substance called glucosylceramide accumulates in macrophages present in certain organs due to the lack of functional GCase. The disorder is hereditary and presents in various subtypes. Freeline is focused on Type 1 Gaucher Disease, which impacts the health of the spleen, liver, lungs and bones. The current standard of care is intravenous infusion of enzyme replacement therapy (ERT) every two weeks, which is a significant treatment burden on the patient.  The aim of Freeline’s investigational gene therapy programme is to deliver a one-time treatment of a long lasting gene therapy for Type 1 Gaucher Disease patients that will provide a consistent and therapeutically relevant level of endogenous GCase, thus eliminating the need for ERT. 

Freeline’s preclinical data to be presented at EWGGD shows that a single infusion of FLT200 results in sustained GCase expression in the liver and greater bioavailability in the bloodstream as compared to ERT. Data collected from a combination of in vitro and in vivo experiments demonstrates that liver expression of GCase results in sustained blood levels of GCase and that the enzyme is taken up by the macrophages in target organs affected by Type 1 Gaucher Disease. FLT200 showed superior levels of GCase uptake in the target organs including spleen, liver, lungs and bone marrow compared to the leading ERT, velaglucerase alfa (VPRIV®).  

Professor Amit Nathwani, Founder and Chief Scientific Officer of Freeline and Professor at UCL Cancer Institute said: 

“Freeline’s proprietary gene therapy platform has been used to generate these promising  preclinical results for Gaucher Disease. We look forward to progressing the program into the clinic and hope to give patients whose lives are severely disrupted by Gaucher Disease the potential to live free of the effects of this disease.” 

Chris Hollowood, Executive Chairman of Freeline said:

“We believe there is a significant opportunity for gene therapy to offer functional cures for a wide range of chronic diseases.  Freeline’s technology has already shown clinical benefit to Haemophilia B patients and we look to leverage this same platform, for not just haemophilia, but also for lysosomal storage disorders (LSDs). Gaucher Disease is our second LSD program after Fabry and these preclinical Gaucher Disease data are very encouraging showing that we may be able to demonstrate a clear clinical benefit for these patients.” 

Oral presentation details are as follows:

Presentation title: Liver-directed AAV gene therapy for Gaucher Disease results in sustained Gcase expression and greater bioavailability for macrophage uptake than velaglucerase alfa (n°3)

Presenter: Carlos Miranda

Date: Friday, 5 July, 2019 from 4:00 - 4:15 pm

Location: University Clermont Auvergne: The Faculty of Dentistry of Clermont - Ferrand: 2, rue de Braga - 63100 Clermont-Ferrand, France

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Last Updated: 05-Jul-2019